Our Impact

• Funded the discovery that specialized, circular bunches of DNA can be found in high levels in glioblastoma tumor cells. These pieces of “extrachromosomal DNA” are believed to be major contributors to tumor growth and treatment resistance. This discovery has the potential to significantly change the way we treat glioblastoma, based on where cancer-fueling genes are located.
• Helped fund the discoveries related to EGFR mutations, which are the most common mutations found in glioblastoma and represent a key focus for how new research and treatments can be constructed.
• Funded the discovery of the 1p/19q co-deletion, which is the key characteristic for identifying and diagnosing oligodendroglioma.
• Funded research that discovered an innovative approach to deliver, through nanoparticles, a DNA damage repair inhibitor drug into the cerebrospinal fluid of a medulloblastoma mouse model. Delivering the drug through this state-of-the-art technique led to tumor regression in the laboratory models and represents a potential promising new treatment strategy for medulloblastoma patients.
• Funded research helping to advance the field of “liquid biopsy” techniques in brain cancer to use DNA extracted from a patients’ cerebrospinal fluid during routine lumbar punctures as a less-invasive means to diagnose, monitor, and tailor treatment plans for glioblastoma and other tumor types.
• Funded the discovery that giving a type of immunotherapy called “checkpoint inhibitors” to patients prior to surgery and standard of care treatments could help these promising therapies, which are revolutionizing the treatment of many other cancers, work in brain cancers like glioblastoma. 
• Funded research that is helping identify risk factors for glioma brain tumors (oligodendroglioma, astrocytoma, glioblastoma, e.g.), especially hereditary factors that could give rise to gliomas in families.

• Launched the DNA Damage Response Consortium with Yale Cancer Center to rapidly evaluate a new class of promising potential treatments for adult and pediatric patients with brain cancer. The consortium also features world-class research laboratories from St. Jude Children’s Research Hospital, the Mayo Clinic, Dana-Farber Cancer Institute, New York University, University of California, San Francisco, University of Minnesota, and Brigham and Women’s Hospital.
• Provided funding, expertise, and advice to support the COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) consortium. CONNECT is an international consortium focused on developing and testing novel therapies in early phase clinical trials for children and adolescents with high-grade glioma brain tumors, including diffuse intrinsic pontine glioma (DIPG)/Diffuse Midline Glioma (DMG). CONNECT includes 18 international partners designed to provide the most promising therapies to children with the poorest prognosis. Learnings from CONNECT are passed along to larger consortia to help speed up bigger trials.
• Funded the early research that enabled the development of the poliovirus treatment currently being evaluated in clinical trials by Duke University, which was highlighted on “60 Minutes.”
• Funded the early research that allowed MD Anderson Cancer Institute scientists to develop an approach that uses a modified version of the common cold virus to attack glioblastoma cells. This research strategy has been successful in early-phase clinical trials, and continues to be evaluated.
• Funded the early research that helped enable the development of the SurVaxM vaccine for glioblastoma, which is now in a phase II clinical trial and was featured on NBC Nightly News. NBTS’s investment affiliate, The Brain Tumor Investment Fund, has also made an investment in the company developing SurVaxM to support an ongoing phase 2b clinical study of SurVaxM in patients with newly diagnosed GBM, as well as subsequent registration-related work to prepare the company for potential commercialization of this therapy. 
• Funded research and provided critical insights from multiple stakeholders in the brain tumor community, including patients, that helped a biopharmaceutical company successfully develop an effective treatment for patients with grade 2 astrocytomas and oligodendrogliomas. 
• As part of the DNA Damage Response Consortium, are providing support for a new clinical trial at Dana-Farber Cancer Institute evaluating a combination approach for recurrent glioblastoma patients with a DNA Damage Repair inhibitor called olaparib and an immunotherapy called pembrolizumab, along with standard of care chemotherapy. Patients are enrolling now.
• Through the DNA Damage Response Consortium, funded research that identified that the drug, AZD1390, in combination with radiation, has the potential to benefit pediatric patients with high-grade glioma.
• Funded the early research that led to the development of a treatment called ERAS-801 that blocks the most common mutation found in GBM tumors from signaling for the cancer cells to grow. This treatment has been granted Fast Track Designation by the U.S. FDA and is now in a phase I clinical trial. 
• Provided support for the international GBM AGILE clinical trial, a first-of-its-kind trial in brain cancer that evaluates potential new treatments in less time than traditional trials.
• Launched the Brain Tumor Investment Fund to support NBTS’s vision to conquer and cure brain tumors by investing in biotechnology, pharmaceutical, and medical device companies developing products to improve the lives of patients with brain tumors. 
• The Brain Tumor Investment Fund has provided capital to six companies developing products for brain tumor patients, including several already in clinical trials or available commercially.
• Organized two Brain Tumor Clinical Trial Endpoints Workshops with the U.S. FDA in 2014 as leaders of the Jumpstarting Brain Tumor Drug Development Consortium of advocacy organizations. A year later, a new, consensus Brain Tumor Imaging Protocol was developed as a result of these efforts, helping to standardize brain tumor medical imaging across the field to help researchers and trial sponsors use imaging data to move toward new drug approvals faster.
• NBTS sponsored, hosted, and participated in a number of meetings for the Response Assessment in Neuro-Oncology (RANO) Working Group, a collaborative body of experts who develop consensus criteria that trial sponsors can employ to better measure efficacy endpoints. NBTS’s convening power helped the group develop a new, “RANO 2.0: Update to the Response Assessment in Neuro-Oncology Criteria for High- and Low-Grade Gliomas in Adults” in 2023. This new criteria will help advance drug development in the field of neuro-oncology by providing trial sponsors with standardized and reliable criteria to measure clinical response and predict efficacy of novel therapies.

Funding for Research 

• Created the brain tumor community’s only annual advocacy day on Capitol Hill to directly ask members of Congress to support policies that benefit brain tumor research, care, and access. Launched in 2011, Head to the Hill now unites more than 300 volunteer advocates from virtually every state in the U.S. to rally together in the nation’s capital. 
• Helped increase annual federal funding for brain cancer research by 85% since 2011. In Fiscal Year (FY) 2011, the National Institutes of Health (NIH) – the largest funders of brain tumor research in the world – funded $280 million in brain cancer research. Our sustained advocacy efforts to increase NIH funding, and brain tumor funding specifically, has led to the agency now estimating it will fund $518 million in brain cancer research in FY 2025. 
• Successfully lobbied to make pediatric brain tumors an eligible topic for funding in the Department of Defense’s Peer-Reviewed Cancer Research Program (PRCRP) in 2016, after the category had been removed in 2015. Each year since, NBTS has continued to ensure that pediatric brain tumors remain an eligible topic, as well as, beginning in 2017, all brain cancer research categories. NBTS-led advocacy has also helped more than double congressional funding for the PRCRP from $50 million in 2016 to $130 million in 2023. Importantly, the number of grants issued to pediatric brain tumor and all brain cancer projects combined has also risen exponentially from $3.75 million in 2016 to $26.8 million in 2023, as NBTS continues to educate the brain tumor research community on the availability of these grants, urges applications, and provides tips on how to successfully apply. Finally, NBTS has provided volunteer advocates with the opportunity to serve as review panelists for PRCRP’s grant making process, ensuring the patient voice is heard in the research process.

Leading New Government Initiatives & Legislation

• National Brain Tumor Society Chief Executive Officer David Arons served on the Blue Ribbon Advisory panel for vice president Joseph Biden’s National Cancer Moonshot in 2016. This panel of only 28 cancer experts and advocates from around the country recommended a set of 10 requirements to the National Cancer Moonshot to greatly accelerate research toward cancer treatments. Cancer Moonshot funding is now supporting multiple unique efforts that will benefit the brain tumor community, including the NBTS-supported OPTIMUM initiative and NCI-CONNECT program.
• Several members of the NBTS staff and Board of Directors were among a select group of experts asked to participate in the first-ever White House Cancer Moonshot Brain Cancers Forum on Glioblastoma (GBM) & Diffuse Intrinsic Pontine Glioma (DIPG) in 2023.
• NBTS helped successfully advocate for the passage of the 21st Century Cures Act in 2016. NBTS supported the 21st Century Cures Act since early on in its drafting process, providing comments on a number of iterations of the draft legislation and discussing specific language with key congressional staff members. NBTS partnered with other advocacy groups and coalitions to generate broad backing for the package and activated volunteer brain tumor advocates to urge their members of Congress to support the bill. The bill provided much-needed additional funding for, and fostered policy and regulatory innovations in, biomedical research.
• Helped successfully champion the passage of the Childhood Cancer STAR Act in 2018. The STAR Act is the most comprehensive childhood cancer bill in history. It expanded opportunities for childhood cancer research, improved efforts to identify and track childhood cancer incidences, and invested in quality of life for childhood cancer survivors. NBTS also played a role in the Childhood Cancer STAR Reauthorization Act – signed into law in 2023 – which reauthorizes continued implementation of the original bill from FY 2024-2028, and related efforts to launch the Childhood Cancer Data Initiative. 
• NBTS helped persuade the maker of Gleostine (lomustine), a standard of care chemotherapy drug, to rejoin the U.S. Medicare program in 2022, after the company had withdrawn this vital medication from Medicare health plans in 2021 — ultimately ensuring the treatment remains affordable and accessible to a greater number of patients.
• Working alongside the Pediatric Brain Tumor Foundation and the Eliminate Brain Cancer Initiative, led advocacy efforts that secured $4 million in funding from the National Cancer Institute (NCI) for leading brain cancer researchers to study the vast heterogeneity of high-grade gliomas in adult, adolescent and young adult (AYA), and pediatric populations through Project HOPE (High-Grade Glioma-Omics in Pediatric and AYA) and Project GBM CARE (Cellular Analysis of Resistance and Evolution) in 2018. These two collaborative, intersecting, and cutting-edge research projects use single-cell RNA sequencing to learn more about tumor heterogeneity, growth, and evolution.
• Successfully advocated for Congress in 2021 to urge the NCI to make a strategic investment specifically in glioblastoma research, which the agency did with the launch of the Glioblastoma Therapeutics Network (GTN). The GTN is a highly experienced group of leading researchers from across the country charged with driving potential new therapies from pre-clinical development into pilot clinical studies in humans. 

Raising the Profile of Brain Tumors

• Championed the creation of a National “Glioblastoma Awareness Day” in 2019 that has now become an annual commemoration for the entire brain tumor and cancer community.
• Planned and held its first-ever Congressional Briefing on Capitol Hill focused on brain cancer in 2018. The briefing was held to educate and engage a bi-partisan gathering of U.S. Senators and. Representatives, national media, and key staffers.
• Advocated with other groups for a resolution designating an official DIPG Awareness Day in the United States.
• Helped launch the Brain Tumor Patients Bill of Rights in 2020 with collaborators from the International Brain Tumor Alliance, as well as the Glioblastoma Bill of Rights, led by OurBrainBank, along with a dozen other brain tumor organizations.